The year 2012 is expected to be a transformative year for biopharmaceutical company AVI BioPharma Inc. (AVII: Quote), if data related to one of its therapeutic development programs, scheduled to be reported in the coming months, pan out positively.
For readers who are new to AVI BioPharma, here's a brief overview of the company's business and what to expect in the coming months...
AVI BioPharma is engaged in the development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. The company's lead product candidate is Eteplirsen, formerly known as AVI-4658, for Duchenne Muscular Dystrophy, or DMD.
Duchenne Muscular Dystrophy, characterized by muscle weakness and calf enlargement, is a genetic disorder caused by a mutation in the gene that codes for dystrophin, a protein in the muscles. This disorder is terminal, and death usually occurs before the age of 30.
DMD affects mostly boys - one in every 3,500 boys worldwide is born with this disease, but girls are rarely affected. It is estimated that there are roughly 30,000 DMD patients in the U.S., EU and Japan. Currently there is no cure for DMD.
AVI BioPharma's Eteplirsen was given orphan drug status in the U.S. in November 2007 and in Europe in December 2008. The drug candidate also has a fast track status in the U.S.
Eteplirsen is currently under a 24-week phase IIb trial for the treatment of DMD. Dosing in all patients in the trial has been completed and unblinded data are anticipated by end of April.
Earlier in the year, an independent Data Safety Monitoring Board, or DSMB, reviewed the 12-week biopsy data from the phase IIb study of Eteplirsen and determined that it was safe to proceed with the trial as planned, since no safety concerns were identified in the study.
A phase 1b/2 clinical trial of Eteplirsen in the indication of DMD has already been completed in the U.K., where the product candidate demonstrated a favorable therapeutic profile with promising safety, biological and exploratory clinical performance assessments.
AVI BioPharma also has two additional investigational drugs for DMD namely, AVI-5038 and an unnamed compound, both under preclinical testing. The company has a collaboration in place with Children's National Medical Center in Washington, D.C. and the Carolinas Medical Center for the development of these two compounds for DMD.
The other investigational drugs in the company's pipeline are AVI-6002 for the treatment of Ebola virus and AVI-6003 for the treatment of Marburg virus, both of which are under phase I testing.
Ebola and Marburg viruses cause hemorrhagic fevers, which are potentially life-threatening infections, marked by severe bleeding, organ failure and a high fatality rate. There are also concerns that these lethal viruses can be used by terrorists and rogue states as a biological weapon of terror.
AVI-6002 and AVI-6003 are being evaluated by the company under a Department of Defense contract managed by the Joint Project Manager Transformational Medical Technologies Project Management Office.
The company's biodefense program against Ebola and Marburg viruses has almost $300 million in potential funding from the U.S. government.
Last month, AVI BioPharma announced positive safety results from phase I single ascending dose studies of AVI-6002 and AVI-6003 for the treatment of Ebola virus and Marburg virus, respectively. Both the trials are progressing as planned to multiple ascending dose studies following the DSMB's recommendation as no safety concerns were identified. The multiple ascending dose studies of AVI-6002 and AVI-6003 are set to begin in the third quarter of this year (3Q).
A quick look at the company's balance sheet...
Since inception in 1980, AVI BioPharma has incurred losses, and has not generated any material revenue from product sales to date.
The net loss in 2011 shrunk to $2.3 million or $0.02 per share from $32.2 million or $0.29 per share in 2010. The annual revenue increased to $47.0 million from $29.4 million in 2010 as a result of increased revenue from the current government contracts for Ebola and Marburg.
At year-end 2011, AVI BioPharma had zero debt and about $40 million in cash and cash equivalents.
For 2012, the company anticipates revenue to range between $40 million and $50 million and operating loss to range between $30 million and $35 million.
AVI BioPharma went public in June 1997, offering its shares at $9 each. The company, which was issued a NASDAQ notice of minimum bid price non-compliance on December 14, 2011, regained compliance with the listing rule last month.
AVII has thus far hit a 52-week low of $0.50 and a 52-week high of $1.98. The stock ended Friday's trading 2.88% higher at $1.07. Year-to-date, AVII is up an impressive 43%.
by RTT Staff Writer
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